This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle…
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem…
The purpose of this study is testing two different Janus kinase (JAK) inhibitors, Abrocitinib and Ritlecitinib, to see if either or both can preserve insulin production in people recently diagnosed with type 1 diabetes (T1D). Researchers believe JAK inhibitors may be able to calm the immune system's attack on insulin producing…